NCT00006057
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
-
- Mount Sinai School of Medicine New York, United States
Clinical trial condition
Lysosomal Storage Diseases, Nervous System
Explore clinical trials studying Lysosomal Storage Diseases, Nervous System. Study availability and eligibility vary by location and protocol.
512 public trials
105 recruiting studies
Lysosomal Storage Diseases, Nervous System trial results
Active, Not Recruiting
A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants
NCT06517914
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
- Locations
-
- CHA Bundang Medical Center Seongnam, Bundang-gu, South Korea
Active, Not Recruiting
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT07082543
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
Active, Not Recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
NCT06150820
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
- Locations
-
- Emory Clinic Atlanta, Georgia, United States
- University of Kansas Medical Center Kansas City, Kansas, United States
- University of Michigan Ann Arbor, Michigan, United States
- Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
Active, Not Recruiting
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
NCT04127578
- Conditions
- Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +20 more
- Locations
-
- Mayo Clinic, 13400 E. Shea Boulevard Scottsdale, Arizona, United States
- Esperanza Clinical, 25220 Hancock Avenue Murrieta, California, United States
- Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510 Englewood, Colorado, United States
- K2 Medical Research, 101 Southhall Lane, Suite 150 Maitland, Florida, United States
Active, Not Recruiting
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
NCT05222906
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
- Locations
-
- Yale University School of Medicine - Investigational Site Number: 8400003 New Haven, Connecticut, United States
- University of Iowa - Investigational Site Number: 8400002 Iowa City, Iowa, United States
- Texas Oncology - Medical City Dallas Site Number : 8400008 Dallas, Texas, United States
- Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 Fairfax, Virginia, United States
Active, Not Recruiting
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
NCT02843035
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Yale University School of Medicine Site Number : 840002 New Haven, Connecticut, United States
- Baylor Institute of Metabolic Diseases Site Number : 840001 Dallas, Texas, United States
- Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 840003 Fairfax, Virginia, United States
- Investigational Site Number : 276001 Mainz, Germany
Active, Not Recruiting
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
NCT04020055
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
- Locations
-
- Emory University Atlanta, Georgia, United States
- The Cleveland Clinic Cleveland, Ohio, United States
- Lysosomal and Rare Disorders Research and Treatment Center, Inc Fairfax, Virginia, United States
- Royal Melbourne Hospital Parkville, Victoria, Australia
Active, Not Recruiting
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
NCT03737214
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
- University of California Irvine Irvine, California, United States
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
Active, Not Recruiting
Early Check: Expanded Screening in Newborns
NCT03655223
- Conditions
- 17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
- Locations
-
- RTI International Research Triangle Park, North Carolina, United States